A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

• Status: Completed
• Trial end date: 2017-08-17
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH). Treatment duration will range from a minimum of approximately 2 months to 6 months per subject. A subject may receive a minimum of one dose level or up to a maximum of 5 dose levels, in sequentially increasing dose strengths. Each dose level will last 28 days.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Millendo Therapeutics, Inc.
• Treatments: Epinephrine; Epinephryl borate; Racepinephrine

Criteria

Inclusion Criteria:

• Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone

• Biochemical marker of disease status of 17-hydroxyprogesterone ≥ 4 times the upper limit of normal

• Chronic glucocorticoid replacement therapy for at least 6 consecutive months

• Stable glucocorticoid and mineralocorticoid regimen for at least 1 month

Exclusion Criteria:

• Non-classic CAH

• Other causes of adrenal insufficiency

• Surgery within the previous 3 months prior to screening or planned surgery during study participation

• History of active cancer requiring medical or surgical therapy within the past 6 months